This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

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Entresto (a tablet comprising a combination of the drugs sacubitril and valsartan), is in clinical development for people with chronic heart failure with preserved ejection fraction. Heart Failure (HF) is a complex clinical syndrome of symptoms and signs that suggest the efficiency of the heart as a pump is impaired. Symptoms include breathlessness and fatigue, and signs of the condition include swollen ankles and crackling sounds in the lungs. People with HF often have a poor quality of life, and about a third of people experience severe and prolonged depressive illness. About half of people with HF have a preserved ejection fraction (HF-PEF), meaning that over 50% of the blood in the left ventricle is pumped out with each contraction of the heart.

Angiotensin II acetate is in clinical development for the treatment of catecholamine and/or vasopressin resistant hypotension due to distributive shock. Shock is a critical condition in which blood pressure drops so low that the brain, kidneys and other vital organs cannot receive enough blood flow to function properly. Distributive shock is the most common type of shock that is most commonly caused by bacterial or fungal infection (septic shock) or allergic reaction (anaphylactic shock). Severe shock is usually treated with drugs called catecholamines or vasopressin. Shock that does not respond to treatment with these drugs is resistant shock.

Udenafil works by blocking the enzyme phosphodiesterase type 5 which regulates the breakdown of ‘cyclic guanine monophosphate’ (cGMP) in the blood vessels of the lungs. Increased levels of cGMP leads to dilation (widening) of the vessels improving blood flow to the lungs, reducing the burden on the heart and improving oxygen supply to the blood. This is expected to improve the symptoms of the condition and may offer an additional treatment option for adolescents (12-18 years old) for the management of congenital heart disease after Fontan palliation who currently have few effective therapies available.

The current standard of care for patients with hypercholesterolaemia is primarily statins which are capable of reducing LDL-C. There is however a subset of patients who are unable to tolerate statins due to adverse effects. Bempedoic acid monotherapy or in combination with ezetimibe are once-daily tablets in development for patients unable to tolerate statins at all, or are not able to tolerate the necessary dose of statin required to reach their LDL-C goal, or are recommended not to use them due to various circumstances. Bempedoic acid and ezetimibe both act in complementary ways to lower LDL-C. These therapies may offer additional and effective treatment options to use in combination with dietary changes and other lipid-modifying therapies to treat primary hypercholesterolaemia or mixed dyslipidaemia.

Bempedoic acid lowers LDL-C via a different mechanism of action and offers the potential advantage of reduced muscular adverse effects when compared to statins which are the current standard of care. Bempedoic acid is being developed for patients at high cardiovascular risk who are unable to reach LDL-C goals with the maximum tolerated dose of statins. The effect of bempedoic acid is additive—not redundant—to that of statins, and if licensed, may offer additional and effective treatment option to use in combination with dietary changes and other lipid-modifying therapies to treat primary hypercholesterolaemia or mixed dyslipidaemia.

Beraprost modified release, in addition to treprostinil, is in clinical development for the oral treatment of pulmonary arterial hypertension (PAH), a rare condition causing high blood pressure in the lungs. PAH can worsen over time and cause several problems including heart failure and blood clots. In PAH, the arteries in the lungs become thickened and …

Bempedoic acid and bempedoic acid combined with ezetimibe are once-daily tablets in development to treat primary hypercholesterolaemia or mixed dyslipidaemia in high risk and very high risk patients. These are patients who cannot reach the target LDL-C goals despite being treated with the maximum tolerated doses of other LDL-C lowering treatment such as statins. Bempedoic acid and ezetimibe both act in different but complementary ways to lower LDL-C. These therapies may offer additional and effective treatment options to use in combination with dietary changes and other lipid-modifying therapies to treat primary hypercholesterolaemia or mixed dyslipidaemia.

Ticagrelor is an oral blood thinning medicine used together with aspirin to prevent problems caused by blood clots and hardening of the arteries that leads to heart attacks or strokes. Ticagrelor is in a class of medications called antiplatelet medications that help prevent blood clots from forming. It is used in adults with acute coronary syndrome, a group of conditions in which blood flow in the vessels supplying the heart is blocked so heart tissue cannot work properly or dies, and which includes heart attack and chest pain. If licensed, ticagrelor will offer an additional antiplatelet therapy for the prevention of CAD in patients with type 2 diabetes.

Tafamidis is an oral (taken by mouth) drug that has the potential to slow the formation of the amyloid deposits that can produce heart problems in people with ATTR‐CM. Tafamidis works by specifically interfering with the disease process that leads to the formation and deposition of the amyloids within the heart tissues. If approved, tafamidis will become the first medicinal treatment option specifically indicated for treating ATTR‐CM, in patients that currently have very limited options available and that mostly focus on symptom management.

Rivaroxaban is an oral medicine which prevents the development of blood clots by blocking the formation of a molecule called thrombin which is a key part of the process of blood clot formation. Rivaroxaban has the potential to reduce thrombin and blood clot formation in people with heart failure and coronary artery disease. This is potentially important as patients with heart failure after an episode of acute decompensation have been seen to have increased levels of thrombin. No other drug is currently licenced for use in this group of patients which targets thrombin.